EMA releases draft guidance on gene therapies

The European Medicines Agency has released draft guidelines to help facilitate the development of gene therapies for a three-month public consultation.

The document aims to support and facilitate developing gene therapies by giving guidance on the types of evidence they should generate to support a marketing authorization application. Gene therapies are different to conventional medicines as they consist of genetic material that needs to be delivered to cells. Many gene therapy developers are at small companies or are academics, and have a lesser familiarity with the regulatory environment than those at big companies.

It follows the structure of the common technical document, the document that must be completed at the time of submission of a marketing authorization application, and provides detailed guidance on scientific and development aspects of gene therapies such as good manufacturing practices.